Specific Aims: During the past 20 years, new treatments have been developed for sarcoidosis.1 Many of these treatments have been directed towards patients with advanced disease. However, the natural course of patients with advanced disease. The proposed registry would identify patients with advanced sarcoidosis. The purpose of the registry is to determine the incidence and prevalence of these forms of the disease in sarcoidosis clinics. In addition, the registry would note the other organ involvement and treatment used for these patients.
Background and significance: The clinical outcome of sarcoidosis is variable. While over half of patients have resolution of their disease within two years of diagnosis, at least a quarter of patients will have chronic disease requiring therapy for more than five years.2;3 Certain manifestations of sarcoidosis are associated with chronic disease. These include pulmonary fibrosis, neurologic and cardiac disease, lupus pernio, and those requiring treatment.4;5 Recent genetic markers have been associated with advanced sarcoidosis.6;7 Most of these studies are based on evaluating patients at single time points. In addition, they lack control sarcoidosis patients who have been matched for race, age, and gender. The use of potent agents such as infliximab have proved useful in treating advanced sarcoidosis.8;9 This treatment regimen is associated with significant cost and requires careful monitoring.10 Other potent agents have also been used for advanced sarcoidosis, including adalimumab, rituximab, cyclophosphamide, and Acthar.11-13 This registry will determine the risk factors for chronic disease and natural course of these manifestations.
Preliminary studies: To date, most of the studies have examined the manifestations of sarcoidosis versus a one time clinical evaluation. These studies have identified that some genes were associated with a good prognosis 14 or chronic disease.15 Serum biomarkers have also been correlated with different clinical outcomes.16 However, the markers are only useful for a limited portion of the sarcoidosis population.
Experimental Design: The primary objective of the study will be to determine the clinical course of various manifestations of advanced sarcoidosis.Secondary objectives will be to compare clinical and genetic markers of advanced versus resolved sarcoidosis. Quality of life for the two groups will also be assessed using the sarcoidosis assessment tool (SAT) and Kings Sarcoidosis Questionnaire (KSQ).